A Double-blind, Escalating Dose, Randomized, Placebo-controlled Study Assessing PK, Safety, Tolerability in Non-ambulant DMD Subjects

• Register: ClinicalTrials.gov
• Last refreshed on: 16 December 2017
• Main ID: NCT01128855
• Date of registration: 20/05/2010
• Prospective Registration: Yes
• Primary sponsor: GlaxoSmithKline
• Public title: A Double-blind, Escalating Dose, Randomized, Placebo-controlled Study Assessing PK, Safety, Tolerability in Non-ambulant DMD Subjects DEMAND I
• Scientific title: A Double-blind, Escalating Dose, Randomized, Placebo-controlled Study to Assess the Pharmacokinetics, Safety and Tolerability of Single Subcutaneous Injections of GSK2402968 in Non-ambulant Subjects With Duchenne Muscular Dystrophy
• Date of first enrolment: July 12, 2010
• Target sample size: 20
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT01128855
• Study type: Interventional
• Study design: Allocation: Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: Triple (Participant, Care Provider, Investigator).
• Phase: Phase 1

Countries of recruitment

France, United States

Contacts

• Name: GSK Clinical Trials
• Affiliation: GlaxoSmithKline

Key inclusion & exclusion criteria

Inclusion Criteria:

- Duchenne muscular dystrophy resulting from a mutation in the DMD gene, confirmed by a
sponsor approved DNA diagnostic technique covering all DMD gene exons, including but
not limited to MLPA (Multiplex Ligation-dependent Probe Amplification), CGH
(Comparative Genomic Hybridisation), SCAIP (Single Condition Amplification/Internal
Primer) or H-RMCA (High-Resolution Melting Curve Analysis), and correctable by
treatment with GSK2402968.

- Age 9 years old or greater at Screening;

- Male;

- Non-ambulant (at least 1 year in a wheelchair) within the last 4 years;

- Life expectancy at least three years;

- Willingness and ability to comply with all protocol requirements and procedures;

- QTc <450msec (based on single or average QTc value of triplicate ECGs obtained over a
brief recording period). Note: QTc may be either QTcB or QTcF, machine read or manual
overread;

- Subjects must be willing to use adequate contraception (condoms or abstinence), from
Screening until at least 5 months after the last dose of study drug;

- Informed assent and/or consent in writing signed by the subject and/or parent(s)/legal
guardian (according to local regulations).

Exclusion Criteria:

- Any additional mutation (such as an additional missing exon for DMD) that cannot be
treated with GSK2402968;

- Current or history of liver or renal disease;

- Acute illness within 4 weeks of anticipated administration of study medication, which
may interfere with study assessments;

- Use of anticoagulants, antithrombotics or antiplatelet agents, previous treatment with
investigational drugs, idebenone or other forms of Coenzyme Q10, within 6 months of
the first administration of study medication;

- Start of glucocorticosteroids within 6 months or non-stable use of
glucocorticosteroids within 3 months of the anticipated first administration of study
medication;

- Positive hepatitis B surface antigen (HbsAg), hepatitis C antibody test (HCV), or
human immunodeficiency virus (HIV) test at Screening;

- Symptomatic cardiomyopathy;

- Use of alcohol from Screening through to the 1 month Follow-up visit ;

- Any Child in Care.



Age minimum: 9 Years
Age maximum: N/A
Gender: Male

Health Condition(s) or Problem(s) studied

Muscular Dystrophies

Intervention(s)

Drug: 6 mg/kg GSK2402968

Drug: 9 mg/kg GSK2402968

Drug: 3 mg/kg GSK2402968

Other: Placebo

Drug: 12 mg/kg GSK2402968

Primary Outcome(s)

Primary Pharmacokinetic Variables:AUC, Cmax,t-max, CL/F [Time Frame: 35 days]

Incidence of Adverse Events [Time Frame: 35 days]

Incidence of Injection Site Reactions [Time Frame: 35 days]

Secondary ID(s)

114118

Source(s) of Monetary Support

Please refer to primary and secondary sponsors